Methods for treating subjects with Prader-Willi syndrome or Smith-Magenis syndrome
Provided are immediate or prolonged administration of certain potassium ATP (K ATP ) channel openers, optionally in combination with growth hormone, to a subject to achieve novel pharmacodynamic, pharmacokinetic, therapeutic, physiological, metabolic and compositional outcomes in the treatment of diseases or conditions involving K ATP channels. Also provided are pharmaceutical formulations, methods of administration and dosing of K ATP channel openers that achieve these outcomes and reduce the incidence of adverse effects in treated individuals. Further provided are methods of co-administering K ATP channel openers with other drugs (e.g., in combination with growth hormone) to treat diseases of humans and animals (e.g., Prader-Willi Syndrome (PWS), Smith-Magenis syndrome (SMS), and the like.
1. A method of reducing hyperghrelinemia in a subject having Prader-Willi syndrome (PWS) or Smith-Magenis syndrome (SMS), comprising administering to said subject for at least 10 weeks a pharmaceutical formulation comprising an effective amount of a K ATP channel opener,
and wherein the method does not comprise administering human growth hormone to the subject.
2. The method of claim 1 , wherein the K ATP channel opener is diazoxide, or a pharmaceutically acceptable salt thereof.
3. The method of claim 2 , wherein the pharmaceutically acceptable salt is diazoxide choline.
4. The method of claim 1 , wherein the pharmaceutical formulation is administered once per day.
5. The method of claim 1 , wherein the pharmaceutical formulation is administered twice per day.
6. The method of claim 1 , wherein the pharmaceutical formulation comprises at least one excipient that affects the rate of release of the K ATP channel opener or the pharmaceutically acceptable salt thereof.
7. The method of claim 1 , wherein the pharmaceutical formulation comprises at least one excipient that delays release of the K ATP channel opener or the pharmaceutically acceptable salt thereof.
8. The method of claim 1 , wherein the pharmaceutical formulation comprises at least one other active ingredient.
9. The method of claim 1 , wherein the pharmaceutical formulation is administered for 1 or more years.
10. The method of claim 1 , wherein the pharmaceutical formulation is administered orally.